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  4. Drug Trials Snapshots: AUGTYRO
  1. Development & Approval Process | Drugs

Drug Trials Snapshots: AUGTYRO

HOW TO USE THIS SNAPSHOT
The information provided in Snapshots highlights who participated in the key clinical trials that supported the original FDA approval of this drug, and whether there were differences among sex, race, age, and ethnic groups. The “MORE INFO” bar shows more detailed, technical content for each section. The Snapshot is intended as one tool for consumers to use when discussing the risks and benefits of the drugs.

LIMITATIONS OF THIS SNAPSHOT
Do not rely on Snapshots to make decisions regarding medical care. Always speak to your healthcare provider about the benefits and risks of a drug.

Some of the information in this Snapshot is for presentation purposes and does not represent the approved conditions of use of this drug. Refer to the AUGTYRO Prescribing Information for all the approved conditions of use of this drug (e.g., indication(s), population(s), dosing regimen(s), safety information).

Snapshots are limited to the information available at the time of the original approval of the drug and do not provide information on who participated in clinical trials that supported later approvals for additional uses of the drug (if applicable).

AUGTYRO (repotrectinib)
Aug-TYE-ro
BRISTOL MYERS SQUIBB CO
Approval date: November 15, 2023


DRUG TRIALS SNAPSHOT SUMMARY:

What is the drug for?

AUGTYRO is a drug used to treat adult patients with advanced or metastatic ROS1-positive non-small cell lung cancer (NSCLC).

How is this drug used?

AUGTYRO is a capsule taken by mouth at 160 mg once daily for 14 days, then increased to 160 mg twice daily, with or without food.

Who participated in the clinical trials?

The FDA approved AUGTYRO based on evidence from one clinical trial (NCT03093116/TRIDENT-1) in adult patients with NSCLC that had changes in the ROS1 gene. The trial was conducted at 152 sites in the United States, the United Kingdom, Canada, the European Union, and Asia (China, Japan, South Korea, Singapore, Hong Kong, and Taiwan). There were 154 patients treated in the United States and 290 treated outside the United States.

The safety of AUGTYRO was evaluated in 351 patients who received at least one dose at the recommended phase 2 dose in TRIDENT-1. The median age of patients who received AUGTYRO was 56 years (range: 20 to 93); 59.5% female; 46.2% Asian, 43% White, 2.6% Black, 0.6% Native Hawaiian or other Pacific Islander, 0.3% American Indian or Alaska Native, 6.3% race not reported, and 0.9% unknown. Some of these patients provided data for the assessment of the benefits of AUGTYRO for ROS1-positive NSCLC.

The efficacy of AUGTYRO was evaluated in two groups of patients in the TRIDENT-1 trial:

  • There were 71 patients in the group of patients with ROS1-positive NSCLC who had not received a prior drug that inhibits ROS1. Characteristics of this group are as follows: median age was 57 years (range: 28 to 80); 60.6% female; 67.6% Asian, 25.4% White, 1.4% Black or African American; and 4.2% Hispanic or Latino.
  • There were 56 patients in the group of patients with ROS1-positive NSCLC who had received one prior ROS1-targeted therapy but had not received prior platinum-based chemotherapy or immunotherapy. Characteristics of this group are as follows: median age was 57 years (range: 33 to 78); 67.9% female; 48.2% Asian, 44.6% White, 1.8 % Black or African American; and 1.8 % Hispanic or Latino.

How were the trials designed?

The benefits and side effects of AUGTYRO for patients with NSCLC that are ROS1-positive were evaluated in one clinical trial. Some patients were previously treated with a drug that inhibits ROS1 for their ROS1-positive NSCLC (pretreated subgroup) and some had not received prior treatment with a drug that inhibits ROS1 (treatment-naïve subgroup). All patients received AUGTYRO by mouth once daily for 14 days, then increased to 160 mg twice daily until either cancer progression or intolerable side effects.

The benefit of AUGTYRO was evaluated by measuring the percentage of patients who had complete or partial shrinkage of their tumors (overall response rate or ORR) and by measuring the duration of that benefit (duration of response or DOR).


DEMOGRAPHICS SNAPSHOT

The pooled safety population for this NDA includes 351 adult patients with advanced solid tumors harboring changes (rearrangements) in the ALK, ROS1, or NTRK1-3 genes who received AUGTYRO.

Figure 1 summarizes how many male and female patients were enrolled in the clinical trial used to evaluate the efficacy of AUGTYRO.

Figure 1. Baseline Demographics by Sex

Pie chart summarizing how many male and female patients were in the clinical trial. In total, 142 (40%) male patients and 209 (60%) female patients participated in the clinical trial.

Source: Adapted from FDA Review

Figure 2 summarizes the percentage of patients by race enrolled in the clinical trial used to evaluate the efficacy of AUGTYRO.

Figure 2. Baseline Demographics by Race

Pie chart summarizing how many White, Black or African American, Asian or Pacific Islander, other, and not reported patients were in the clinical trial. In total, 151 (43%) White patients, 9 (3%) Black or African American patients, 162 (46%) Asian or Pacific Islander patients, 7 (2%) other patients, and 22 (6%) not reported patients participated in the clinical trial.

Source: Adapted from FDA Review

Figure 3 summarizes the percentage of patients by age enrolled in the clinical trial used to evaluate the efficacy of AUGTYRO.

Figure 3. Baseline Demographics by Age

Pie chart summarizing how many patients by age were in the clinical trial. In total, 256 (73%) patients between 18 and 65 years of age, 72 (20%) patients between 65 and 75 years of age, and 23 (7%) patients 75 years of age and older participated in the clinical trial.

Source: Adapted from FDA Review

Figure 4 summarizes the percentage of patients by ethnicity enrolled in the clinical trial used to evaluate the efficacy of AUGTYRO.

Figure 4. Baseline Demographics by Ethnicity

Pie chart summarizing how many Hispanic, not Hispanic, and unknown patients were in the clinical trial. In total, 10 (3%) Hispanic or Latino patients, 328 (93%) not Hispanic or Latino patients, and 13 (4%) unknown patients participated in the clinical trial.

Source: Adapted from FDA Review

What are the benefits of this drug?

In the trial, 79% of the 71 patients with ROS1-positive NSCLC who were ROS1 inhibitor naïve experienced complete or partial shrinkage of their tumors; 91% of these patients had complete or partial shrinkage of their tumors which lasted more than six months.

In the trial, 38% of the 56 patients with ROS1-positive NSCLC who were pretreated with an ROS1 inhibitor experienced complete or partial shrinkage of their tumors which lasted more than six months for 80% of patients.

Were there any differences in how well the drug worked in clinical trials among sex, race, and age?

  • Sex: AUGTYRO worked similar in males and females.
  • Race: AUGTYRO worked similar in Asian, Black or African American, and White patients.
  • Age: AUGTYRO worked similarly in patients younger and older than 65 years of age.

What are the possible side effects?

AUGTYRO may cause serious side effects including brain and nervous system effects, lung injury, liver injury, muscle aches and increased enzymes, increased uric acid, bone fractures, and harm to unborn babies.

The most common side effects of AUGTYRO are dizziness, changes to taste, numbness or tingling in extremities, constipation, shortness of breath, unstable gait, fatigue, slow thinking, and muscular weakness.

Were there any differences in side effects among sex, race and age?

  • Sex: The majority of patients were female. The number of male patients was limited; therefore, differences in the occurrence of side effects among male patients compared to female patients could not be determined.
  • Race: The majority of patients were White. The number of patients in other races were limited; therefore, differences in in the occurrence of side effects among races could not be determined.
  • Age: The majority of patients were younger than 65 years of age; therefore, differences in the occurrence of side effects among younger and older age groups could not be determined.

GLOSSARY

CLINICAL TRIAL: Voluntary research studies conducted in people and designed to answer specific questions about the safety or effectiveness of drugs, vaccines, other therapies, or new ways of using existing treatments.
COMPARATOR: A previously available treatment or placebo used in clinical trials that is compared to the actual drug being tested.
EFFICACY: How well the drug achieves the desired response when it is taken as described in a controlled clinical setting, such as during a clinical trial.
PLACEBO: An inactive substance or “sugar pill” that looks the same as, and is given the same way as, an active drug or treatment being tested. The effects of the active drug or treatment are compared to the effects of the placebo.
SUBGROUP: A subset of the population studied in a clinical trial. Demographic subsets include sex, race, and age groups.

PRESCRIBING INFORMATION

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